While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, revealed persistent concerns regarding the durability of these treatments and their potential liver toxicity.

The transition from complex and costly ex vivo strategies to platforms that enable direct cellular intervention within the body, known as in vivo therapies, is marking a paradigm change in the field of gene and cell therapies by simplifying manufacturing, improving tissue targeting and expanding clinical access to treatments.

The pathogenesis of multiple sclerosis (MS) has been tied to ineffective immune control of Epstein-Barr virus-driven autoimmune responses. Patients with MS are deficient in protective adaptive natural killer cells (pNK cells) in contrast to healthy individuals. These pNK cells are positive for NKG2A, NKG2C and NKG2D and recognize and kill autoreactive B cells in a selective and efficient manner.

As the many challenges facing cell therapies are being addressed, the CAR T field continues to evolve beyond its original design of T cells engineered to target hematological malignancies. During the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, several studies showed how this technology is being redefined as programmable and adaptable immune cells with expanded functional versatility.

Researchers from Onco3r Therapeutics BV presented preclinical data on O3R-5671, a novel salt-inducible kinase 3 (SIK3) inhibitor developed for the treatment of inflammatory bowel disease and other autoimmune diseases.

Recent evidence in atopic dermatitis (AD) points to the involvement of additional pro-inflammatory pathways besides core Th2 responses. Current therapeutic approaches that work target mostly the IL-4/IL-13 pathway, but the duration of response and depth could be improved.